Drug policy at the margins: the case of growth hormone replacement for adults with severe growth hormone deficiency

Wendy L Lipworth, Ken Ho, Ian H Kerridge and Richard O Day
Med J Aust 2012; 197 (4): 204-205. || doi: 10.5694/mja11.11572

Are current standards of evidence always appropriate when making the decision to subsidise treatment?

The Pharmaceutical Benefits Scheme (PBS) provides the Australian community with subsidised access to medicines. Decisions about which medicines are listed on the PBS are made by the Minister for Health and Ageing, on the advice of the Pharmaceutical Benefits Advisory Committee (PBAC) — an independent statutory body charged with assessing the clinical benefit and cost-effectiveness of the medicines under consideration.

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  • Wendy L Lipworth1
  • Ken Ho2
  • Ian H Kerridge3
  • Richard O Day4

  • 1 Australian Institute of Health Innovation, University of New South Wales, Sydney, NSW.
  • 2 Centres for Health Research, Princess Alexandra Hospital, Brisbane, QLD.
  • 3 Centre for Values, Ethics and the Law in Medicine, University of Sydney, Sydney, NSW.
  • 4 Clinical Pharmacology and Toxicology, St Vincent's Hospital, Sydney, NSW.


Competing interests:

Ken Ho has been on an Eli Lilly advisory board and has been a consultant to Pfizer. Richard Day has provided occasional advice to Merck Australia about rofecoxib and etoricoxib.

  • 1. Molitch ME, Clemmons DR, Malozowski S, et al. Evaluation and treatment of adult growth hormone deficiency: an Endocrine Society clinical practice guideline. J Clin Endocrinol Metab 2011; 96: 1587-1609.
  • 2. Woodhouse LJ, Mukherjee A, Shalet SM, et al. The influence of growth hormone status on physical impairments, functional limitations, and health-related quality of life in adults. Endocr Rev 2006; 27: 287-317.
  • 3. Ho KK; GH Deficiency Consensus Workshop Participants. Consensus guidelines for the diagnosis and treatment of adults with GH deficiency II: a statement of the GH Research Society in association with the European Society for Pediatric Endocrinology, Lawson Wilkins Society, European Society of Endocrinology, Japan Endocrine Society, and Endocrine Society of Australia. Eur J Endocrinol 2007; 157: 695-700.
  • 4. National Institute for Clinical Excellence. Human growth hormone (somatropin) in adults with growth hormone deficiency. Technology Appraisal 64. London: NICE, 2003 (reviewed 2006). (accessed May 2012).
  • 5. Pharmaceutical Benefits Advisory Committee. July 2011 PBAC Outcomes — 1st time decisions not to recommend. Canberra: Department of Health and Ageing, 2011. (accessed Oct 2011).
  • 6. Underwood LE, Attie KM, Baptista J; Genentech Collaborative Study Group. Growth hormone (GH) dose-response in young adults with childhood-onset GH deficiency: a two-year, multicenter, multiple-dose, placebo-controlled study. J Clin Endocrinol Metab 2003; 88: 5273-5280.
  • 7. Hoffman AR, Kuntze JE, Baptista J, et al. Growth hormone (GH) replacement therapy in adult-onset GH deficiency: effects on body composition in men and women in a double-blind, randomized, placebo-controlled trial. J Clin Endocrinol Metab 2004; 89: 2048-2056.
  • 8. Carroll PV, Christ ER, Bengtsson BA, et al. Growth hormone deficiency in adulthood and the effects of growth hormone replacement: a review. Growth Hormone Research Society Scientific Committee. J Clin Endocrinol Metab 1998; 83: 382-395.
  • 9. Attanasio AF, Lamberts SW, Matranga AM, et al. Adult growth hormone (GH)-deficient patients demonstrate heterogeneity between childhood onset and adult onset before and during human GH treatment. Adult Growth Hormone Deficiency Study Group. J Clin Endocrinol Metab 1997; 82: 82-88.
  • 10. Pharmaceutical Benefits Advisory Committee. Guidelines for preparing submissions to the PBAC – December 2008. Canberra: Department of Health and Ageing, 2008. (accessed May 2012).


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